Mass生物实验室的环孢素类神经保护剂获治疗ALS药物专利权

雪茄客

Maas Biolab received United States Patent 7,446,093, European Union Patent 1,091,750 and Patents inIndia, Canada and New Zealandfor its Mitogard® cyclosporin formulas. Mitogard® is designed for central nervous system delivery to treat neurodegenerative diseases, in particular amyotrophic lateral sclerosis (ALS). The patent covers the use of cyclosporin in DMSO formulations for neuroprotection and immunosuppression via cerebrospinal fluid (CSF), vascular, nasal and pulmonary delivery. The 19 European countries include Germany, France, the United Kingdom, Italy, Spain, Netherlands, Sweden and Switzerland.
Cyclosporin formulation is a compounding challenge, since it is water insoluble. The Maas Biolab solution is the cyclosporin-solubilizing excipient dimethyl sulfoxide (DMSO) that has low toxicity, low viscosity for easy syringeability, and chemical compatibility with the components of CSF pumps, ports and catheters. Water-free, full strength, ready-to-infuse Mitogard® has low viscosity and long-term stability within functioning pumps.
Cyclosporin does not cross the intact blood-brain barrier. Neurosurgeon and Maas Biolab CEO Marcus Keep, M.D., created Mitogard® to bypass the blood-brain barrier with direct CSF infusion of cyclosporin to get it into the spinal cords and brains of ALS patients. Cyclosporin is a powerful neuroprotectant in the G93A mouse model of ALS, slowing loss of motor neurons and paralysis. Long-term therapy with very low-dose CSF cyclosporin is not expected to be systemically immunosuppressive nor nephrotoxic. Maas Biolab has FDA Orphan Drug Designation for cyclosporin treating ALS, and is conducting NIH-funded animal toxicity trials of Mitogard®.
Maas Biolab, LLC
Maas Biolab is a private pharmaceutical company in Albuquerque, New Mexico, USA, developing Mitogard®, the only cyclosporin formula designed for CSF delivery, to treat amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. (http://www.maasbiolab.com)
http://www.swissbiotechassociation.ch/html/e/index.php?1=1&id=306856

菡萏尘雾

Mass生物实验室的新药Mitogard®（环孢素类配方）获得美国（7446093号）、欧盟（1091750号）、印度、加拿大等国的专利权。Mitogard®被设计为通过中枢神经系统给药，专门用于治疗神经系统变性疾病，特别是肌萎缩侧索硬化症（ALS）。专利涵盖环孢素的两种用法，分别是：1、在二甲基亚砜中添加环孢素作为神经保护剂；2、通过脑脊液、血管、鼻腔、肺部给药，作为免疫抑制剂。19个欧洲国家包括德国、法国、英国、意大利、西班牙、荷兰、瑞典、瑞士。

环孢素因其不溶于水使得用药方案存在多重挑战。Mass生物实验室的解决方案是以低毒性、低粘度（易灌注）的二甲基亚砜作为药物辅料以增加环孢素的溶解性，同时提高脑脊液泵、医用导管等组件的化学药剂耐受性。Mitogard®以其无水、全强度、易灌注和粘度低的特性，在功能泵中能保持长期的稳定性。

环孢素并不通过完整的血-脑屏障。神经外科医生、Mass生物实验室首席执行官Marcus Keep博士介绍：可以通过直接的脑脊液输注，使得环孢素绕开血-脑屏障而作用于ALS病人的脊髓和大脑。在G93A ALS老鼠模型中，环孢素是一种强有力的神经保护剂，可以减缓神经元凋亡和瘫痪的进程。长期小剂量使用，预计不会出现系统性的免疫抑制作用和肾中毒。Mass生物实验室环孢素对于ALS的治疗，获得了FDA孤儿药的认证。现阶段，Mitogard®正在进行国家卫生研究院资助的动物毒性试验。

Maas生物有限公司是位于美国新墨西哥州阿尔伯克基的一家私营制药公司。他们开发的药物，是唯一以环孢素为配方，通过脑脊液给药，治疗肌萎缩侧索硬化症（ALS）和其他神经变性疾病的药物。

（感谢晨雾大哥、雪茄客、小房子夫妇对翻译的帮助）

miwu

我翻阅了这个公司网站上关于这个药的全部新闻，我个人的理解是：该药的实验从2007年开始，目前完成了三期的临床前实验（动物），看来效果不错，可以延长生命，所以获得了欧美一些国家的专利权。目前准备进入人体的临床试验。部分翻译如下：
新闻发布时间：2007年10月 11日
ALBUQUERQUE, N.M.--(BUSINESS WIRE)--Maas BiolAB received a Small Business Innovation Research (SBIR) grant of $2.1 million over three years from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) for the translational project “Intrathecal Cyclosporin for the Treatment of Amyotrophic Lateral Sclerosis (ALS).”Mass Biolab公司从国家神经疾病和中风协会得到$2100000的资金以进行跨度为三年的使用内鞘环孢霉素治疗als的实验。
Cyclosporin, a direct mitochondrial neuroprotectant, substantially extends life in ALS mice. Mitogard®, the only cyclosporin formula designed for cerebrospinal fluid administration, was developed because systemic cyclosporin does not easily reach the brain and spinal cord. Dose escalation toxicology and pharmacokinetics safety studies of intrathecal Mitogard® will be performed under Good Laboratory Practices (GLP) by Northern Biomedical Research, enabling Investigational New Drug (IND) approval for ALS clinical trials.环孢霉素是一种直接线粒体神经保护剂，可以延长患有als老鼠的寿命。Mitogard，是仅有的专为管理脑脊髓流速设计的环孢霉素配方，因为现有的环孢霉素药物无法进入大脑和脊髓。该药物的毒性、药物代谢动力安全实验将由Northern Biomedical Research进行，以使该药获得als临床实验的批准。

新闻发布时间：2008年12月3日
经过第一年的安全实验，我们高兴地宣布，我们将进行深入的药物毒性实验。接下来的两年我们将进行二期实验，即进行进一步的临床前的药物稳定性、毒性、药物代谢动力实验。

菡萏尘雾

miwu姐辛苦了，自身的负担已经很重了，还为大家发布最新的咨询，不甚感激！

菡萏尘雾

Maas Biolab currently is conducting animal studies to assess the safety of the compound
Mass生物试验室目前在进行动物实验以评估该化合物的安全性。
人体实验，估计不远了！

旅途中的候鸟

:time::victory: